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Glutaric acidemia IIa

MONDO:0700073

Any multiple acyl-CoA dehydrogenase deficiency in which the cause of the disease is a mutation in the ETFA gene.

Also known as: ETFA deficiency, GA2A, glutaric acidemia 2A, multiple acyl-CoA dehydrogenase deficiency caused by mutation in ETFA

20 clinical trials for this condition and its sub-types.

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Broader categories

Cardiovascular disorder (1051) Disease (680) Heart disorder (300) Metabolic disease (233) Musculoskeletal system disorder (207) Inherited lipid metabolism disorder (189) Hereditary disease (176) Cardiomyopathy (144) Hypertrophic cardiomyopathy (112) Inborn mitochondrial metabolism disorder (58)
Trials to join now! 10 Not yet recruiting 1 Not yet finished but already full! 6 Completed 2 Terminated 1
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  • New drug aims to tame hard-to-control seizures in rare mitochondrial disorders

    Disease control Terminated

    This study tested a drug called vatiquinone in 68 people with mitochondrial disease and epilepsy that doesn't respond to standard treatments. Participants were randomly assigned to receive either vatiquinone or a placebo for 24 weeks to see if the drug could reduce the number of …

    Phase: PHASE2, PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control

    Last updated Jun 27, 2026 12:03 UTC

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